RARECast

548 Avsnitt

1. A Heart Attack that Led to a Rare Disease Diagnosis

   Publicerades: 2025-01-30
2. Targeting Rare Liver Diseases with Gene Editing Therapies

   Publicerades: 2025-01-23
3. Using AI to De-Risk Rare Disease Drug Development

   Publicerades: 2025-01-16
4. Addressing a Growing Demand for Plasma-Derived Therapies

   Publicerades: 2025-01-09
5. Empowering Patients with Data to Drive Drug Development

   Publicerades: 2025-01-02
6. How Advocates Are Advancing a Treatment for an Ultra-Rare Disease

   Publicerades: 2024-12-26
7. Engineering B Cells to Produce Therapeutic Proteins

   Publicerades: 2024-12-19
8. A Pipeline Win as BridgeBio Learns to Balance Theory with Practice

   Publicerades: 2024-12-12
9. Considering What It Will Take for Payers to Embrace Advances in Sequencing

   Publicerades: 2024-12-05
10. Finding Strength

    Publicerades: 2024-11-27
11. A Non-Profit Works to Advance a DMD Gene Therapy

    Publicerades: 2024-11-21
12. Accepting What We Can’t Understand

    Publicerades: 2024-11-14
13. A Gene Therapy Company Born from a Father’s Efforts to Save his Daughter

    Publicerades: 2024-11-07
14. Lowering Barriers to Clinical Trial Participation with Do-It-Yourself Blood Sample Collection

    Publicerades: 2024-10-31
15. Scaling N-of-1 Therapies to Viability

    Publicerades: 2024-10-24
16. Building Gene Therapies that Address Gain and Loss of Function Simultaneously

    Publicerades: 2024-10-17
17. How One Woman Climbed from Patient to Advocate

    Publicerades: 2024-10-10
18. Shortening the Diagnostic Odyssey

    Publicerades: 2024-10-03
19. Making Exomes More Revealing

    Publicerades: 2024-09-26
20. How One Patient Organization Catalyzed Drug Development

    Publicerades: 2024-09-19